Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges
EA Taha, J Lee, A Hotta - Journal of Controlled Release, 2022 - Elsevier
The discovery of clustered regularly interspaced short palindromic repeats (CRISPR)
genome editing technology opened the door to provide a versatile approach for treating …
genome editing technology opened the door to provide a versatile approach for treating …
Viral Vectors for the in Vivo Delivery of CRISPR Components: Advances and Challenges
M Asmamaw Mengstie - Frontiers in Bioengineering and …, 2022 - frontiersin.org
The Clustered Regularly Interspaced Short Palindromic Repeat (CRISPR) and its
accompanying protein (Cas9) are now the most effective, efficient, and precise genome …
accompanying protein (Cas9) are now the most effective, efficient, and precise genome …
Familial hypercholesterolemia: global burden and approaches
L Tokgozoglu, M Kayikcioglu - Current cardiology reports, 2021 - Springer
Abstract Purpose of Review Familial hypercholesterolemia (FH) is the most common genetic
metabolic disorder characterized by markedly elevated LDL-C levels from birth leading to …
metabolic disorder characterized by markedly elevated LDL-C levels from birth leading to …
Viral vectors and extracellular vesicles: innate delivery systems utilized in CRISPR/Cas-mediated cancer therapy
Gene editing-based therapeutic strategies grant the power to override cell machinery and
alter faulty genes contributing to disease development like cancer. Nowadays, the principal …
alter faulty genes contributing to disease development like cancer. Nowadays, the principal …
[HTML][HTML] Advancing targeted protein degradation for metabolic diseases therapy
QQ Zhou, HT **ao, F Yang, YD Wang, P Li… - Pharmacological …, 2023 - Elsevier
The development and application of traditional drugs represented by small molecule
chemical drugs and biological agents, especially inhibitors, have become the mainstream …
chemical drugs and biological agents, especially inhibitors, have become the mainstream …
Evolving AAV-delivered therapeutics towards ultimate cures
X He, BA Urip, Z Zhang, CC Ngan, B Feng - Journal of Molecular Medicine, 2021 - Springer
Gene therapy has entered a new era after decades-long efforts, where the recombinant
adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene …
adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene …
[HTML][HTML] Exosome-based Ldlr gene therapy for familial hypercholesterolemia in a mouse model
Z Li, P Zhao, Y Zhang, J Wang, C Wang, Y Liu… - Theranostics, 2021 - ncbi.nlm.nih.gov
Familial hypercholesterolemia (FH), with high LDL (low-density lipoprotein) cholesterol
levels, is due to inherited mutations in genes, such as low-density lipoprotein receptor …
levels, is due to inherited mutations in genes, such as low-density lipoprotein receptor …
Current approaches in CRISPR-Cas9 mediated gene editing for biomedical and therapeutic applications
A single gene mutation can cause a number of human diseases that affect the quality of life.
Until the development of clustered regularly interspaced short palindromic repeats …
Until the development of clustered regularly interspaced short palindromic repeats …
The potential of CRISPR/Cas9 gene editing as a treatment strategy for inherited diseases
Clustered regularly interspaced short palindromic repeats (CRISPR) is a promising
innovative technology for genomic editing that offers scientists the chance to edit DNA …
innovative technology for genomic editing that offers scientists the chance to edit DNA …
Familial Hypercholesterolemia: JACC Focus Seminar 4/4
J Brandts, KK Ray - Journal of the American College of Cardiology, 2021 - jacc.org
Detecting familial hypercholesterolemia (FH) early and “normalizing” low-density lipoprotein
(LDL) cholesterol values are the 2 pillars for effective cardiovascular disease prevention in …
(LDL) cholesterol values are the 2 pillars for effective cardiovascular disease prevention in …